Efficacy and tolerability of old and new drugs used in the treatment of immune thrombocytopenia: Results from a long-term observation in clinical practice

Abstract

BackgroundMany patients with immune thrombocytopenia (ITP) may require special attention and long-term treatment. Little is known on the efficacy and tolerability of the drugs used in practice.Material and methodsWe retrospectively reviewed the results of therapy of 400 patients with chronic ITP. All Patients were treated at our institution between 1996–2016 under consideration of guidelines, general recommendations, and individual aspects, including gender, age, weight, comorbidity, patient’s medical history and bleeding risk.ResultsTreatment was not required in 25% of patients (n = 100) during observation. In treated patients (n = 300), the rate of patients that responded and tolerated treatment with prednisolone was 59% (52/88), with azathioprine 32% (29/90), with eltrombopag 49% (31/63), with romiplostim 59% 27/45, with IVIG (intravenous immunoglobulines) 75% (94/126), with anti-D 37% (19/52) and with dexamethasone 60% (25/42) patients. Eighteen treated patients (6%) entered sustained remission after treatment with various drugs. Twenty-six patients underwent splenectomy (Splx) resulting in sustained remission in 15 cases (60%). Only two patients remained refractory to Splx and to all used drugs.DiscussionNone of the currently available drugs used in the treatment of ITP are invariably safe and effective. Responses, the duration of response, intolerability, and the course of disease are unpredictable. Although the treatment of ITP has considerably improved in the recent years, the currently available drugs may rarely cure affected patients. The need for safe and effective therapy in ITP is evident. Optimal treatment decisions for each patient remains a challenge in many cases.

Treatments for Primary Immune Thrombocytopenia: A Review

“…It is clear that corticosteroids, and more specifically, high-dose dexamethasone, are an effective initial treatment for ITP. The side effect profile of corticosteroids, including weight gain, hypertension, and diabetes, can be an issue for some patients, but corticosteroids are still a generally safe and easily administered treatment, making them an appropriate first-line choice [11].…”

“…Anti-RhD can be useful in conjunction with corticosteroids in patients who are RhD positive. One study reports an efficacity of 50-70% in patients, while others report 37% efficacy [11,12]. However, side effects such as severe hemolysis, nausea, fever, headache have been reported in some patients, so care should be taken [11,12].…”

“…Some studies show a 65-70% complete response (defined as the absence of significant bleeding) with a 60-70% long-term response [12,16,17]. One retrospective study found that 60% of patients had sustained remission after splenectomy [11]. Another retrospective study of 174 patients showed that 88% of patients had a good response after therapy, but 20% of those patients relapsed.…”

“…A study in France reported a 40% response rate, 29% of those with a persistent response after discontinuing azathioprine [26]. Another retrospective study of 96 patients found that 54% of patients responded to azathioprine, though only 2% had a sustained response [11]. Leukopenia and increases in transaminases were reported, as well as alopecia, gastrointestinal effects, and increased risk of malignancy, specifically lymphoma [24,25].…”

<p>Clinical Phenotype and Response to Different Lines of Therapy in Elderly with Immune Thrombocytopenia: A Retrospective Study</p>

“… 12 , 13 They appear to offer many advantages over other drugs in current use, especially in the older population. 14 , 15 However, within the context of clinical practice, potential limitations in selecting a treatment are represented by current gaps in knowledge of the management of persistent or chronic ITP.…”